Results 101 to 110 of about 266,379 (285)

Suppression of HBV replication by the expression of nickase- and nuclease dead-Cas9

open access: yesScientific Reports, 2017
Complete removal of hepatitis B virus (HBV) DNA from nuclei is difficult by the current therapies. Recent reports have shown that a novel genome-editing tool using Cas9 with a single-guide RNA (sgRNA) system can cleave the HBV genome in vitro and in vivo.
Takeshi Kurihara   +12 more
doaj   +1 more source

Exon 7 splicing of ERα predicts poor prognosis and increases phenotypic heterogeneity in luminal a subtype breast cancer

open access: yesFEBS Open Bio, EarlyView.
ERα splice variant ERα∆7 lacks the C‐terminus, and its expression may change phenotypes of breast cancers. Our results showed that ERα∆7 is found in the luminal A subtype, and elevated ERα∆7 levels are linked to improved cell survival with lower proliferation and migration.
Long Wai Tsui   +10 more
wiley   +1 more source

Integrate CRISPR/Cas9 for protein expression of HLA-B*38:68Q via precise gene editing. [PDF]

open access: yes, 2019
The determination of null- or low-expressed HLA alleles is clinically relevant in both hematopoietic stem cell transplantation and solid organ transplantation.
Reed, Elaine F   +2 more
core   +1 more source

MiR‐513a promotes human erythroid differentiation by modulating c‐Jun

open access: yesFEBS Open Bio, EarlyView.
During early human erythropoiesis, miR‐513a promoted erythroid differentiation in primary human CD34+ hematopoietic stem‐progenitor cells and human TF‐1 erythroleukemic cells by indirectly decreasing c‐Jun and phospho‐c‐Jun expression, which are associated with increased GATA1 expression.
MinJung Kim   +11 more
wiley   +1 more source

Large‐scale bidirectional arrayed genetic screens identify OXR1 and EMC4 as modifiers of αSynuclein aggregation

open access: yesFEBS Open Bio, EarlyView.
Activation of the mitochondrial protein OXR1 increases pSyn129 αSynuclein aggregation by lowering ATP levels and altering mitochondrial membrane potential, particularly in response to MSA‐derived fibrils. In contrast, ablation of the ER protein EMC4 enhances autophagic flux and lysosomal clearance, broadly reducing α‐synuclein aggregates.
Sandesh Neupane   +11 more
wiley   +1 more source

Exploratory Analysis of ELP1 Expression in Whole Blood From Patients With Familial Dysautonomia

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Background Familial dysautonomia (FD) is a hereditary neurodevelopmental disorder caused by aberrant splicing of the ELP1 gene, leading to a tissue‐specific reduction in ELP1 protein expression. Preclinical models indicate that increasing ELP1 levels can mitigate disease manifestations.
Alejandra González‐Duarte   +13 more
wiley   +1 more source

CRISPR-Cas revolution in Agriculture: From precision genome editing to sustainable crop improvement

open access: yesCurrent Innovation in Agriculture Science
The CRISPR-Cas system has emerged as a transformative tool in agricultural biotechnology, revolutionizing the landscape of crop improvement. This review paper explores the multifaceted applications of CRISPR technology in agriculture, from its ...
Anantha Rama A
doaj  

Efficient CRISPR-rAAV engineering of endogenous genes to study protein function by allele-specific RNAi. [PDF]

open access: yes, 2015
Gene knockout strategies, RNAi and rescue experiments are all employed to study mammalian gene function. However, the disadvantages of these approaches include: loss of function adaptation, reduced viability and gene overexpression that rarely matches ...
Dowdy, Steven F   +5 more
core   +1 more source

Developmental and Epileptic Encephalopathy due to Biallelic Pathogenic Variants in PIGM

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective PIGM encodes a critical enzyme in the glycosylphosphatidylinositol (GPI)‐anchor biosynthesis pathway. While promoter‐region mutations in PIGM have been associated with a relatively mild phenotype characterized by portal vein thrombosis and absence seizures, recent evidence suggests that coding‐region mutations result in a more severe
Júlia Sala‐Coromina   +11 more
wiley   +1 more source

Cas9+ conditionally-immortalized macrophages as a tool for bacterial pathogenesis and beyond. [PDF]

open access: yes, 2019
Macrophages play critical roles in immunity, development, tissue repair, and cancer, but studies of their function have been hampered by poorly-differentiated tumor cell lines and genetically-intractable primary cells.
Barton, Gregory M   +7 more
core  

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